Promising new drug tames rare blood cancer in early trial
NCT ID NCT03580655
First seen Apr 25, 2026 · Last updated May 17, 2026 · Updated 4 times
Summary
This study tested a drug called avapritinib in 107 people with advanced systemic mastocytosis, a rare and serious blood disorder. The drug targets a specific genetic change that drives the disease. Results showed that many participants experienced significant improvement or remission of their symptoms, though ongoing treatment is needed to maintain control.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno
Salerno, 84131, Italy
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Azienda Ospedaliero-Universitaria Careggi, CRIMM - Centro di Ricerca ed Innovazione per le Malattie Mieloproliferative
Florence, 50134, Italy
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Beatson West of Scotland Cancer Centre - NHS Greater Glasgow and Clyde
Glasgow, G12 0YN, United Kingdom
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CHU Toulouse - Hôpital Larrey
Toulouse, 31059, France
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Centro Ricerche Cliniche di Verona - Azienda Ospedaliera Universitaria Integrata di Verona
Verona, 37134, Italy
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Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital
London, SE1 9RT, United Kingdom
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Herbert Irving Comprehensive Cancer Center
New York, New York, 10032, United States
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Huntsman Cancer Institute
Salt Lake City, Utah, 84112, United States
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Hôpital Necker-Enfants Malades
Paris, 75015, France
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Klinik und Poliklinik für Innere Medizin III, Klinikum rechts der Isar der TU München
Munich, 81675, Germany
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Mays Cancer Center
San Antonio, Texas, 78229, United States
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Medizinische Universität Wien, Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie
Vienna, 1090, Austria
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Odense University Hospital, Department of Haematology
Odense, DK-5000, Denmark
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Oslo University Hospital-Rikshospitalet, Hematology
Oslo, 0372, Norway
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Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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St. Michael's Hospital
Toronto, Ontario, M5B 1W8, Canada
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Stanford Cancer Institute
Stanford, California, 94305, United States
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The University of Kansas Cancer Center
Westwood, Kansas, 66205, United States
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Uniklinik RWTH Aachen, Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltranslplantation
Aachen, 52074, Germany
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University Medical Center Groningen (UMCG)
Groningen, 9713 GZ, Netherlands
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Pennsylvania, Abramson Cancer Center
Philadelphia, Pennsylvania, 19104, United States
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University of Texas, MD Anderson Cancer Center
Houston, Texas, 77030, United States
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Universitätsklinikum Hamburg-Eppendorf, Zentrum für Onkologie
Hamburg, 20246, Germany
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Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, lnternistische Onkologie, Hämostaseologie
Leipzig, 04103, Germany
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Universitätsmedizin Mannheim III. Medizinische Klinik
Mannheim, 68167, Germany
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Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu, Klinice Hematologii, Nowotworów Krwi i Transplantacji Szpiku
Wroclaw, 50-367, Poland
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Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii
Gdansk, 80-214, Poland
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo
Toledo, 45071, Spain
Conditions
Explore the condition pages connected to this study.